Short-term Clinical Study of CN128 in Thalassemia Patients
Status:
Completed
Trial end date:
2019-12-17
Target enrollment:
Participant gender:
Summary
1. Primary objectives:
- To study the tolerance and safety of multiple oral administration of CN128 in
patients with thalassemia aged 16 years and above.
- To study the pharmacokinetics of CN128 in thalassemia patients aged 16 and above by
multiple oral administrations of CN128
2. Design:
The study is designed as a safety, tolerability and pharmacokinetic parameters study,
phase Ib trial.
The study is consisted of: multiple dose tolerance and safety study; multiple
administration pharmacokinetics.
3. Subject inclusion criteria:
- Thalassemia patients with serum ferritin ≥ 500 µg/L
- Patients aged 16 and above
- HB≥80 g/L before administration
- Voluntarily participate in the experiment, and the process of obtaining informed
consent met the requirements of GCP.
4. Subject exclusion criteria:
- Hepatitis B surface antigen positive, hepatitis B core antibody positive and
HBV-DNA positive, hepatitis C anti-HCV positive, HIV positive, Treponema pallidum
positive
- History of active digestive tract diseases (including gastric ulcer, duodenal
ulcer, gastroesophageal varices, ulcerative colitis, Crohn's disease, digestive
tract tumors, familial genetic polyps), history of digestive tract perforation,
history of digestive tract surgery and influence on drug absorption, and other
investigators believe that patients with potential intestinal complications
- Liver dysfunction (ALT or AST > 2.5×ULN); or renal dysfunction (serum creatinine >
1.5×ULN)
- Uncontrolled active infections
- Patients currently taking CYP3A strong inducer or inhibitor drugs or drugs that may
prolong the QT interval without temporary suspension of use or temporary
substitution of the said drugs
- ect.
5. Usage:
All subjects fasted prior to administration of study drug using 240 ml warm water. The
people can not drink water within 1h before administration.
6. Pharmacokinetic assessment of CN128 administration:
PK parameters of CN128 include AUC 0-t, AUC 0-∞, Cmax, Tmax, t1/2, CL/F, Vd/ F, MRT, λz,
Css-av, Css-min, Css-max, Accumulation rate, Fluctuation index, etc.
7. Safety and tolerability assessments:
Evaluation was based on the incidence rate of adverse events (AE) after the
administration, study termination information, vital signs, physical examination,
laboratory tests and ECG.
8. Statistics